New enzyme therapy shows promise for rare metabolic disorder in babies
Disease control
Completed
This study tested a weekly injection of pegzilarginase in 3 infants under 2 years old with arginase 1 deficiency, a rare genetic disorder that causes harmful buildup of arginine. The goal was to see if the drug safely lowers arginine levels and helps manage the condition. Because…
Phase: PHASE3 • Sponsor: Immedica Pharma AB • Aim: Disease control
Last updated May 12, 2026 13:41 UTC